EU/3/02/088 - orphan designation for treatment of Pseudomonas aeruginosa lung infection (including colonisation) in cystic fibrosis

Colistimethate sodium

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2011 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.

On 19 February 2002, orphan designation (EU/3/02/088) was granted by the European Commission to Pharmax Limited, United Kingdom, for colistimethate sodium for the treatment of Pseudomonas aeruginosa lung infection (including colonisation) in cystic fibrosis.

Pharmax Limited changed its name to Forest Laboratories UK Ltd in July 2002.

What is Pseudomonas aeruginosa lung infection in cystic fibrosis patients?

Chronic infection of the lung with the bacterium Pseudomonas aeruginosa represents a hallmark of cystic fibrosis. It can induce damage to the lung tissue and respiratory insufficiency, which is life threatening.

What is the estimated number of patients affected by the condition?

At the time of designation, Pseudomonas aeruginosa lung infection in cystic fibrosis affected approximately 1.3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 49,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP).

* The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

Pseudomonas aeruginosa lung infection in cystic fibrosis is treated predominantly with antibiotic therapy administered by a variety of routes, oral, intravenous and as an aerosol via nebulisation. Several antibiotics had been authorised for the condition in some countries in the Community, at the time of submission of the application for orphan designation. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that colistimethate sodium might be of potential significant benefit for the treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis, particularly in terms of its low potential for resistance and its ease of administration as a dry powder inhalation.

How is this medicine expected to work?

Colistimethate is an antibiotic produced by certain strains of bacteria. The sponsor has developed a formulation for dry powder inhalation to be administered with a commercial device. In contrast to many antibiotics, which target specific molecules in the bacteria, colistimethate is claimed to have a “physical” mechanism of action disrupting the bacterial membrane. This action cannot be countered by the resistance mechanisms employed by bacteria against other antibiotics with specific cellular enzyme targets, resulting in a very low degree of resistance to colistimethate.

What is the stage of development of this medicine

At the time of the submission of the application for orphan designation clinical trials with colistimethate sodium dry powder inhaler in cystic fibrosis patients with Pseudomonas aeruginosa lung infection had not been initiated.

Colistimethate sodium dry powder inhaler had not been marketed anywhere worldwide for this condition or designated as an orphan medicinal product elsewhere, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 December 2001 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the Community) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/02/088: Public summary of positive opinion for orphan designation of colistimethate sodium for the treatment of Pseudomonas aeruginosa lung infection (including colonisation) in cystic fibrosis

AdoptedReference Number: EMEA/COMP/75/02 Rev. 3

Key facts

• Active substance:
  Colistimethate sodium
• Intended use:
  Treatment of Pseudomonas aeruginosa lung infection (including colonisation) in cystic fibrosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/02/088
• Date of designation:
  19/02/2002
• Sponsor:

  Forest Laboratories UK Ltd; Riverbridge House; Crossways Business Park; Dartford; Kent DA2 6SL; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]